Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
Official Title
A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies
Acronym
Not provided
Organization
Barbara Ann Karmanos Cancer InstituteOTHER
Status Module
Record Verification Date
Feb 2016
Overall Recruitment Status or Expanded Access Status
No longer available
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jan 2007
Primary Completion Date
Jan 2015Actual
Completion Date
Jan 2015Actual
First Submitted Date
Jan 16, 2007
First Submission Date that Met QC Criteria
Jan 16, 2007
First Posted Date
Jan 18, 2007Estimated
Results Waived
Not provided
Results First Submitted Date
Not provided
Results First Submitted that Met QC Criteria
Not provided
Results First Posted Date
Not provided
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Feb 26, 2016
Last Update Posted Date
Feb 29, 2016Estimated
Sponsor/Collaborators Module
Responsible Party, by Official Title
Voravit Ratanatharathorn, Principal Investigator, Barbara Ann Karmanos Cancer InstitutePrincipal Investigator
Lead Sponsor
Barbara Ann Karmanos Cancer InstituteOTHER
Collaborators
Name
Class
National Cancer Institute (NCI)
NIH
Oversight Module
No data available
No data is available for this block.
Description Module
Brief Summary
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and mycophenolate mofetil before the transplant may stop this from happening.
PURPOSE: This clinical trial is studying how well umbilical cord blood stem cell transplant works in treating patients with hematologic cancer or other disease.
Detailed Description
OBJECTIVES:
Primary
Determine the efficacy of double umbilical cord blood stem cell transplantation using a conditioning regimen comprising lower doses of busulfan and fludarabine phosphate and low-dose total body irradiation, in terms of stem cell engraftment at 60 days post transplantation, in patients with hematologic cancer or other diseases.
Determine the merits of conducting a larger, comparative study of this regimen.
Secondary
Determine mortality within 100 days of transplantation in these patients.
OUTLINE: This is a pilot study.
Reduced-intensity conditioning regimen: Patients receive busulfan IV over 3 hours on days -9 to -8 and fludarabine phosphate IV on days -7 to -3. Patients then undergo low-dose total body irradiation on day 0.
Graft-versus-host disease prophylaxis: Patients receive tacrolimus IV twice daily and mycophenolate orally or IV three times daily beginning on day -3.
CNS prophylaxis and/or treatment: Patients with a history of CNS involvement receive prophylactic cytarabine (Ara-C) intrathecally (IT) prior to transplant. Patients also undergo lumbar puncture (LP) to test for active CNS disease. Patients with cerebrospinal fluid positive for leukemia receive Ara-C IT every 2-3 days until a repeat LP shows no remaining leukemic cells. Three days after the last LP and after one final dose of Ara-C, patients begin the conditioning regimen.
Double umbilical cord blood (UCB) donor stem cell transplantation (SCT): Patients undergo double UCB donor SCT on day 0.
PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study.
Conditions Module
Conditions
Leukemia
Lymphoma
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Precancerous Condition
Secondary Myelofibrosis
Keywords
accelerated phase chronic myelogenous leukemia
blastic phase chronic myelogenous leukemia
childhood chronic myelogenous leukemia
relapsing chronic myelogenous leukemia
adult acute lymphoblastic leukemia in remission
recurrent adult acute lymphoblastic leukemia
childhood acute lymphoblastic leukemia in remission
recurrent childhood acute lymphoblastic leukemia
adult acute myeloid leukemia in remission
adult acute myeloid leukemia with 11q23 (MLL) abnormalities
adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with t(15;17)(q22;q12)
adult acute myeloid leukemia with t(16;16)(p13;q22)
adult acute myeloid leukemia with t(8;21)(q22;q22)
Design Module
Study Type
Expanded Access
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Not provided
Interventional Study Design
No data available
No data is available for this block.
Biospecimen
No data available
No data is available for this block.
Enrollment
Not provided
Arms/Interventions Module
Arm Groups
Not provided
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Busulfan
Drug
3 mg/kg intravenously over 3 hours
Busulfex®
Myleran®
Cytarabine
Drug
Patients with previous history of CNS involvement will receive pre-transplant intrathecal Cytarabine (Ara-C) (30 mg/M2) therapy.
DepoCyt(TM)
Liposomal Ara-C
Fludarabine phosphate
Drug
25 mg/M2/day IV
Fludara
mycophenolate mofetil
Drug
Orally at the dose of 1 gm every 8 hours.
Cellcept
tacrolimus
Drug
0.015 mg/kg IV every 12 hours by continuous infusion.
Outcomes Module
No data available
No data is available for this block.
Eligibility Module
Eligibility Criteria
DISEASE CHARACTERISTICS:
Histologically confirmed diagnosis of 1 of the following:
Acute myeloid leukemia meeting the following criteria:
M0-M7 histologic subtypes by French-American-British classification
Previously treated disease
Meets 1 of the following criteria:
Persistent disease as evidenced by 5-30% persistent blasts in bone marrow after induction or salvage therapy
In second or subsequent complete remission (CR)
In first CR with 1 of the following high-risk features:
Philadelphia chromosome present
Noncore-binding factor type of chromosomal abnormalities
Myelodysplastic syndromes with 1 of the following International Prognostic Scoring System (IPSS) scores:
Intermediate-1
Intermediate-2
High-risk score with transfusion dependence
Chronic myelogenous leukemia meeting 1 of the following criteria:
In accelerated or blastic phase
Failed prior imatinib mesylate therapy
Acute lymphoblastic leukemia meeting 1 of the following criteria:
In first CR with any of the following high-risk features:
Philadelphia chromosome present
Translocation t(4;11) present
WBC > 30,000/mm³ (adult patients)
More than 4 weeks from initiation of treatment was required to achieve CR (adult patients)
DNA index of near haploid (N=23 chromosomes) (pediatric patients)
In second or subsequent CR
Persistent disease as evidenced by 5-20% persistent blasts in bone marrow after induction or salvage therapy
Hodgkin's or non-Hodgkin's lymphoma meeting the following criteria:
Recurrent or refractory disease
Tumor ≤ 5 cm in diameter
Myeloma or plasma cell neoplasm meeting 1 of the following staging criteria:
Stage III at presentation
Stage I-II at presentation
Not responding OR progressed after first-line therapy
Chronic lymphocytic leukemia or Waldenstrom's macroglobulinemia with refractory or progressive disease after first-line therapy
No 5-6/6 HLA-matched related or 7-8/8 HLA-matched unrelated marrow or peripheral blood stem cell donor available
No single 4-6/6 HLA-A, -B, or -DRB1-matched umbilical cord blood unit ≥ 3.5 x 10^7 nucleated cells/kg available
PATIENT CHARACTERISTICS:
ECOG performance status (PS) 0-2 OR Karnofsky or Lansky PS 70-100%
Not pregnant
Fertile patients must use effective contraception prior to and during study participation
HIV negative
Bilirubin < 3.0 mg/dL
AST and ALT ≤ 3 times upper limit of normal
Creatinine < 2.0 mg/dL OR creatinine clearance > 50 mL/min
Cardiac ejection fraction > 50% by echocardiogram OR shortening fraction > 27%
No uncontrolled symptomatic congestive heart failure
No unstable angina pectoris
No cardiac arrhythmia
FEV_1 > 50% of normal
Forced vital capacity > 50% of normal
DLCO normal
Oxygen saturation > 92% on room air (for patients < 5 years of age)
No history of allergic reactions attributed to compounds of similar chemical or biological composition to busulfan and fludarabine phosphate
No ongoing or active infection
No psychiatric illness or social situation that would preclude study compliance
No other uncontrolled illness
PRIOR CONCURRENT THERAPY:
See Disease Characteristics
At least 4 weeks since prior and no concurrent surgery
At least 4 weeks since prior and no other concurrent investigational or commercial agents or therapies for the malignancy, including chemotherapy, biologic therapy, or radiotherapy